PYRUKYND gets EU approval for thalassaemia treatment

The European Commission has granted marketing authorization for PYRUKYND (mitapivat), an oral pyruvate kinase activator, for treating anemia in adults with transfusion-dependent and non-transfusion-dependent alpha- or beta-thalassaemia.

Agios Pharmaceuticals Inc. (NASDAQ: AGIO) developed the treatment, which will be commercialized and distributed in Europe by Avanzanite Bioscience under an exclusive agreement signed in June 2025. The drug received orphan medicinal product designation.

The approval marks PYRUKYND's second indication in the EU, following its 2022 approval for adults with pyruvate kinase deficiency. The European Medicines Agency Committee for Medicinal Products for Human Use had previously issued a positive opinion supporting the authorization.

The approval is based on results from two global, randomized, double-blind, placebo-controlled Phase 3 trials called ENERGIZE and ENERGIZE-T. The ENERGIZE trial enrolled 194 non-transfusion-dependent patients, while ENERGIZE-T studied 258 transfusion-dependent patients. Both trials evaluated the efficacy and safety of mitapivat 100 mg twice daily compared to placebo.

Thalassaemia is a rare, inherited blood disease affecting hemoglobin production in red blood cells. The condition causes anemia, fatigue and serious complications. Some patients require regular blood transfusions, while others need them only intermittently.

Avanzanite Bioscience, founded in 2022 and based in Amsterdam, operates across 32 European countries. The company focuses on rare disease medicines and has a team of more than 100 professionals.

This information is based on a press release from Avanzanite Bioscience and Agios Pharmaceuticals.

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